NR2E3

▎药明康德内容团队编辑近期，全球细胞和基因疗法（CGT）领域迎来系列进展。其中包括：Moderna与Immatics公司达成18亿美元合作，结合Immatics的T细胞受体平台与Moderna的mRNA技术开发创新抗癌疗法。Ocugen公司可用于治疗多种遗传性视网膜疾病的基因疗法让超过80%患者的视力得到维持或改善。本文将节选其中部分重要进展做简单介绍，仅供读者参阅。图片来源：123RF———✦研发进展✦———超80%患者视力维持或改善，“通用型”基因疗法早期临床结果积极Ocugen公司日前宣布了其创新基因疗法OCU400，在治疗与NR2E3和RHO突变相关的色素性视网膜炎（RP）和携带CEP290基因突变的莱伯先天性黑蒙（LCA）患者的1/2期临床试验中获得积极结果。接受治疗的12名患者中，10名（83%）患者的视力维持稳定或者获得改善。OCU400是基于核激素受体基因NR2E3开发的基因疗法，NR2E3调节视网膜组织内的多种不同生理功能，OCU400可能重置细胞基因网络和细胞稳态，从而具有改善遗传性视网膜疾病患者的视网膜健康和功能的潜力，而不需考虑导致疾病的基因突变种类。基于生物标志物终点支持加速批准，创新基因疗法关键性临床试验设计获FDA认可Rocket Pharmaceuticals公司日前宣布，已经与美国FDA就其治疗达农病（Danon Disease）的基因疗法RP-A501的关键性2期临床试验设计达成一致讨论。这项试验将为单臂、多中心2期临床试验，使用基于生物标志物的共同主要终点，包括LAMP2蛋白的表达水平改善，和左心室体积的缩小。这项研究将支持RP-A501的加速批准。缓解率100%，潜在“first-in-class“CAR-T疗法早期临床结果积极PeproMene Bio公司宣布，靶向B细胞激活因子受体（BAFF-R）的CAR-T疗法PMB-CT01，在治疗复发/难治性B细胞非霍奇金淋巴瘤的1期临床试验中，完成首个剂量组的给药，未发现剂量限制性毒性。接受治疗的3名患者中，总缓解率为100%，包括2名完全缓解和1名部分缓解。这一研究将继续进行下一剂量组的给药。BAFF-R是几乎只在B细胞中表达的BAFF的主要受体。因为BAFF-R介导的信号通路是维持B细胞生存必需的信号通路，并且促进B细胞增殖，因此肿瘤难于通过丢失BAFF-R抗原来逃避免疫反应。这让BAFF-R靶向CAR-T疗法在治疗B细胞血液癌症方面具有显著潜力。CAR-T疗法失效患者获得完全缓解，创新多靶点细胞疗法早期临床结果积极Marker Therapeutics公司宣布，首位接受其在研细胞疗法MT-601治疗的淋巴瘤患者获得完全缓解，这名患者曾经接受过CD19靶向CAR-T疗法后产生疾病复发。MT-601是该公司开发的多肿瘤相关抗原特异性T细胞疗法。这种细胞疗法通过选择性扩增患者体内的肿瘤抗原特异性T细胞生成，没有经过基因工程改造。由于靶向多种肿瘤相关抗原，肿瘤细胞不容易通过丢失单一抗原来逃避疗法的攻击。百吉生物首款TCR-T细胞疗法获批临床，治疗多种晚期实体瘤百吉生物（Biosyngen）日前宣布，其针对晚期肺癌、胃癌等多种实体瘤的TCR-T细胞疗法BRL03的1/2期临床试验IND申请获得美国FDA的许可。BRL03是该公司研发的首款进入临床的TCR-T在研疗法。该公司的IDENTIFIER平台基于来自上千个体的TCR和肿瘤蛋白组数据库，支持抗原、抗体和TCR的发现。BRL03还通过表达其它功能模块组件让T细胞在实体瘤微环境中不易耗竭，从而更持久地发挥效力。体内生成Trop2靶向CAR-T细胞，mRNA疗法完成首例患者给药Myeloid Therapeutics公司宣布，在研疗法MT-302在治疗晚期或转移性上皮肿瘤的1期临床试验中完成首例患者给药。MT-302是该公司的主打体内免疫细胞重编程疗法，它将编码靶向Trop2的嵌合抗原受体的mRNA递送到患者体内，在髓系细胞中选择性表达。这种体内重编程策略的设计旨在给患者提供高度标准化的个体化治疗，消除体外处理患者细胞的复杂程序，降低疗法开发时间和成本。———✦合作、融资、M&A✦———18亿美元，Moderna合作开发创新抗癌疗法Moderna与Immatics公司宣布达成了一项战略性研发合作，为具有高度未竟医疗需求的癌症患者开创全新的变革性疗法。这项合作将Immatics的T细胞受体平台与Moderna的mRNA技术相结合，开发包括双特异性、细胞疗法和癌症疫苗在内的各种治疗模式的创新疗法。在细胞疗法方面，双方计划开展临床前研究和1期临床试验，评估Immatics靶向PRAME的TCR-T细胞疗法IMA203，与Moderna靶向PRAME的mRNA癌症疫苗联用的安全性和有效性。该联合疗法的目的是进一步增强IMA203的T细胞反应。2seventy bio与药明巨诺扩展合作，加快基于T细胞的免疫疗法研发2seventy bio和药明巨诺日前宣布，双方意图扩展其战略研发联盟。扩展的研发合作将添加2seventy bio管线中的两款候选疗法，一款为基于TCR技术的实体瘤疗法，另一款为治疗自身免疫性疾病的CAR-T疗法。双方最初合作开发的靶向MAGE-A4的T细胞疗法项目预计在2023年底进入临床开发阶段。开发吸入式基因疗法，新锐推进新一代慢病毒递送系统AlveoGene公司日前宣布成立，该公司致力于开发吸入式基因疗法，治疗具有未竟医疗需求的呼吸道疾病（不包括囊性纤维化）。该公司的InGenuiTy平台开发的基因疗法基于新一代慢病毒递送系统，可以通过雾化器重复给药，高效转导肺部上皮细胞。这一技术已经有十多年的开发历史，并且得到约7200万英镑研究基金的支持。该公司的首款候选疗法AVG-001是一款用于治疗α1抗胰蛋白酶缺乏症（AATD）的吸入式基因疗法，旨在促进局部α1抗胰蛋白酶的表达，该公司预计在未来2-3年将这款疗法推向临床开发。6000万美元助力家族性神经疾病基因疗法开发，克睿基因和Frametact达成合作克睿基因（Cure Genetics）和Frametact联合宣布，双方达成合作开发和许可协议。将利用克睿基因独有的VELP平台开发创新腺相关病毒载体，治疗家族性神经疾病。根据协议，克睿基因将获得总计6000万美元的前期和里程碑付款。除了上述动态，本周细胞和基因疗法领域还有一些其它进展，限于篇幅，本文不再一一介绍。希望该领域迎来更多的新进展和突破，惠及更多患者。大家都在看作为药明康德旗下专注于细胞和基因疗法的CTDMO，药明生基致力于加速和变革基因和细胞治疗及其他高端治疗的开发、测试、生产和商业化。药明生基能够助力全球客户将更多创新疗法早日推向市场，造福病患。如您有相关业务需求，欢迎点击下方图片填写具体信息。▲如您有任何业务需求，请长按扫描上方二维码，或点击文末“阅读原文/Read more”，即可访问业务对接平台，填写业务需求信息▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料：[1] Coeptis Therapeutics Provides Safety and Dosing Update from Phase 1 Trials Investigating DVX201 in Relapsed/Refractory AML or High Risk MDS and Hospitalized COVID-19 Infection. Retrieved September 14, 2023, from https://www.prnewswire.com/news-releases/coeptis-therapeutics-provides-safety-and-dosing-update-from-phase-1-trials-investigating-dvx201-in-relapsedrefractory-aml-or-high-risk-mds-and-hospitalized-covid-19-infection-301927462.html[2] 2seventy bio and JW Therapeutics Announce Intent To Expand Strategic Partnership to Accelerate the Research and Development of T Cell-based Immunotherapies and Autoimmune Therapies. Retrieved September 14, 2023, from https://www.businesswire.com/news/home/20230912702923/en[3] Affimed Receives FDA Fast Track Designation for AFM13 in Combination with AlloNK® for the Treatment of Patients with Relapsed or Refractory Hodgkin Lymphoma. Retrieved September 14, 2023, from https://www.globenewswire.com/news-release/2023/09/12/2741447/0/en/Affimed-Receives-FDA-Fast-Track-Designation-for-AFM13-in-Combination-with-AlloNK-for-the-Treatment-of-Patients-with-Relapsed-or-Refractory-Hodgkin-Lymphoma.html[4] Marker Therapeutics Announces Complete Response in First Lymphoma Patient Treated with MT-601 after CAR T Relapse. Retrieved September 14, 2023, from https://www.globenewswire.com/news-release/2023/09/11/2740646/0/en/Marker-Therapeutics-Announces-Complete-Response-in-First-Lymphoma-Patient-Treated-with-MT-601-after-CAR-T-Relapse.html[5] Moderna and Immatics Announce Strategic Multi-Platform Collaboration to Develop Innovative Oncology Therapeutics. Retrieved September 14, 2023, from https://www.globenewswire.com/news-release/2023/09/11/2740660/0/en/Moderna-and-Immatics-Announce-Strategic-Multi-Platform-Collaboration-to-Develop-Innovative-Oncology-Therapeutics.html[6] Biosyngen received FDA approval for Phase I/II Clinical Trials for BRL03, targeting Lung Cancer, Gastric Cancer and other advanced Solid Tumors. Retrieved September 14, 2023, from https://www.prnewswire.com/news-releases/biosyngen-received-fda-approval-for-phase-iii-clinical-trials-for-brl03-targeting-lung-cancer-gastric-cancer-and-other-advanced-solid-tumors-301923030.html[7] PeproMene Bio, Inc. Announced Completion of the First Dose Cohort and Opening of the Second Dose Cohort in Its B-cell Non-Hodgkin Lymphoma (B-NHL) Phase 1 Clinical Trial of PMB-CT01 (BAFFR-CAR T Cells). Retrieved September 14, 2023, from https://www.prnewswire.com/news-releases/pepromene-bio-inc-announced-completion-of-the-first-dose-cohort-and-opening-of-the-second-dose-cohort-in-its-b-cell-non-hodgkin-lymphoma-b-nhl-phase-1-clinical-trial-of-pmb-ct01-baffr-car-t-cells-301921330.html[8] Cure Genetics and Frametact Reach a $60 Million Collaboration and Licensing Agreement for the Development of Gene Therapy for Familial Neurological Diseases. Retrieved September 14, 2023, from https://www.prnewswire.com/news-releases/cure-genetics-and-frametact-reach-a-60-million-collaboration-and-licensing-agreement-for-the-development-of-gene-therapy-for-familial-neurological-diseases-301928075.html[9] AlveoGene Launches to Develop Unique Inhaled Gene Therapies for Rare Respiratory Disorders. Retrieved September 14, 2023, from https://www.globenewswire.com/news-release/2023/09/14/2742984/0/en/AlveoGene-Launches-to-Develop-Unique-Inhaled-Gene-Therapies-for-Rare-Respiratory-Disorders.html[10] Rocket Pharmaceuticals Reaches FDA Alignment on Pivotal Phase 2 Trial Design for RP-A501 in Danon Disease. Retrieved September 14, 2023, from https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-reaches-fda-alignment-pivotal-phase-2[11] Ocugen Announces Positive Clinical Study Update from the Phase 1/2 Trial of OCU400, a Modifier Gene Therapy Product Candidate, for the Treatment of Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA). Retrieved September 14, 2023, from https://www.globenewswire.com/news-release/2023/09/13/2742453/0/en/Ocugen-Announces-Positive-Clinical-Study-Update-from-the-Phase-1-2-Trial-of-OCU400-a-Modifier-Gene-Therapy-Product-Candidate-for-the-Treatment-of-Retinitis-Pigmentosa-RP-and-Leber-.html[12] Myeloid Therapeutics Initiates Patient Dosing with MT-302, a Novel TROP2-Targeting RNA CAR, in Phase 1 Study for Advanced or Metastatic Epithelial Tumors. Retrieved September 14, 2023, from https://myeloidtx.com/myeloid-therapeutics-initiates-patient-dosing-with-mt-302/免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

Favorablesafetyandtolerabilitypro >ofOCU400investigational drug productin RP and LCA subjects to date Clinical study update suggests continued positive trends in Best-Corrected Visual Acuity (BCVA) and Multi-Luminance Mobility Testing (MLMT), as well as positive trends in Low-Luminance Visual Acuity (LLVA) among treated eyes 83% (10/12) of subjects demonstrated stabilization or improvement in treated eye either on BCVA or LLVA or MLMT scores from baseline Notably, 86% (6/7) of RHO mutation subjects experienced either stabilization of or increase in MLMT scores from baseline including a subset of 29% (2/7) that demonstrated a 3 Lux luminancelevel improvement MALVERN, Pa., Sept. 13, 2023 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced a clinical study update for Retinitis Pigmentosa (RP) participants treated in the Phase 1/2 trial to assess the safety and efficacy of OCU400 for RP associated with NR2E3 and Rhodopsin (RHO) mutations and Leber congenital amaurosis (LCA) with mutation(s) in the CEP290 gene. This clinical study update is an extension of results provided by Ocugen on April 14, 2023, and includes additional subjects from the high dose group. The Company believes that OCU400—Ocugen’s therapeutic approach, utilizing a proprietary modifier gene therapy platform—has the potential to be a gene-agnostic therapeutic for RP and LCA patients with inherited retinal degeneration. “This clinical study update supports our vision to help change the lives of patients suffering from inherited retinal diseases,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “We remain dedicated to our mission of pioneering breakthroughs in biotechnology and believe that OCU400 has the potential to have an impact on the future treatment of patients with RP and LCA.” This Phase 1/2 trial is a multicenter, open-label, dose ranging study. A total of 18 subjects with vision impairment due to RP associated with RHO and NR2E3 gene mutations received a unilateral subretinal injection of either a low dose (1.66 x 1010 vg/mL), medium dose (3.33 x 1010 vg/mL), or high dose (1.66 x 1011 vg/mL) of OCU400. The study pro a diverse group of subjects aged 18-77 years old, with varied disease stages, racial and ethnic pro medical histories, and mutation subgroups. Ocugen further expanded this Phase1/2 trial to enroll LCA patients with CEP290 gene mutation and pediatric patients with NR2E3, RHO and CEP290 mutations. Inherited retinal diseases (IRDs) such as RP and LCA encompass a group of genetic disorders that affect the retina, the light-sensitive tissue at the back of the eye. These diseases often lead to a gradual loss of vision over time and can ultimately result in blindness. Stabilization of vision is crucial for patients with IRDs due to the progressive and degenerative nature of these conditions. Preserving remaining vision, slowing disease progression, or improving the vision can significantly impact patients’ quality of life. It not only enhances the quality of life for affected individuals but also provides hope for future treatments that may ultimately lead to vision restoration. Comprehensive care, early diagnosis, and access to emerging therapies are essential components of a strategy to stabilize vision in IRD patients. “I am gratified to see the progress we have made in our pursuit of developing a novel gene-agnostic therapy for RP and LCA. Our team's unwavering dedication to advancing modifier gene therapy research demonstrated positive preliminary clinical results that offer renewed hope to patients and their families. We remain resolute in our mission and vision to bring a bright future to those with inherited or age-related retinal diseases through courageous innovation and unwavering determination,” said Dr. Arun Upadhyay, Chief Scientific Officer, Head of Research, Development and Medical at Ocugen. This clinical study update is based on the currently available data from Phase 1 (dose-escalation: Cohort 1, 2 and 3) and the Phase 2 (open enrollment) portion of the study. The exploratory efficacy update includes data for 12 subjects who have completed a minimum of 6-month follow up. The data set comprised of 2 subjects [Cohort 1] with 12-month follow-up, 5 subjects [N=2 from Cohort 1 and N=3 from Cohort 2] with 9-month follow-up, and 5 subjects [N=2 from Cohort 3 and N=3 from Open Enrollment/Phase 2] with 6-month follow-up. “It is an important steppingstone for Ocugen and its mission to help the nearly 1.6 million patients affected by RP and LCA worldwide. For those suffering from these IRDs, this clinical trial update provides hope. It is encouraging to see a favorable safety and tolerability pro positive efficacy readout for OCU400 in RP patients,” said Dr. Lejla Vajzovic, Associate Professor of Ophthalmology with Tenure, Director of Duke Vitreoretinal Fellowship Program at Duke Eye Center and Duke University School of Medicine and leader in gene-therapy research. Key efficacy outcomes from 12 subjects demonstrated: BCVA: 83% (10/12) of subjects demonstrated stabilization or improvements in treated eyes in BCVA scores from baseline 42% (5/12) of OCU400 treated eyes experienced 4-letter improvement and 33% (4/12) treated eyes experienced 7-letter improvement in BCVA from baseline 57% (4/7) of RHO subjects’ treated eyes experienced 4-letter improvement and 43% (3/7) treated eyes experienced 7-letter improvement in BCVA scores from baseline LLVA: 83% (10/12) of subjects demonstrated stabilization or improvement in treated eyes in LLVA scores from baseline 42% (5/12) of OCU400 treated eyes experienced 5-letter improvement (1 line) in LLVA from baseline, with 25% (3/7) increasing by 10 letters (2 lines) 43% (3/7) of RHO subjects experienced 5-letter improvement (1 line) in treated eyes in LLVA scores from baseline, among which 29% (2/7) increased by 10 letters (2 lines) MLMT: 75% (9/12) of subjects demonstrated stabilization or improvement in treated eyes in MLMT scores from baseline 33% (4/12) of subjects in the low, medium, and high dose cohorts experienced at least 1 Lux luminance level improvement from baseline in treated eyes, among which 17% (2/12) increased by 3 Lux luminance levels 86% (6/7) of RHO subjects experienced either stabilization or increases in MLMT scores from baseline, among which 29% (2/7) improved by 3 lux levels “The RHO mutation affects more than 10,000 people in the US,” said Dr. David Birch, Scientific Director, Retina Foundation of the Southwest and Principal investigator of the study. “In my view, the clinical study update supports the gene-agnostic mechanism of action of OCU400 in RHO patients. The improvements in BCVA, LLVA and MLMT in this patient population are very exciting and encouraging because stabilization alone could be considered as a treatment benefit.” The clinical study update from the Phase 1/2 clinical trial demonstrated that OCU400 continued to be generally safe and well-tolerated in subjects across different mutations and dose levels. There were no serious adverse events (SAEs) related to the investigational product in the low and medium-dose cohorts. In the high-dose and open-enrollment cohorts, SAEs were reported for two subjects. Adverse events were mostly deemed related to the surgical procedure and resolved within a few days to weeks. “The clinical study update released by Ocugen appears to have a tangible biological impact on Retinitis Pigmentosa associated with NR2E3 and RHO mutations,” said Dr. David Boyer, Clinical Professor of Ophthalmology USC/Keck School of Medicine Los Angeles, CA and Partner, Retina Vitreous Associates Medical Group. “These findings may indicate a huge step forward in the way we approach and treat this condition. We remain optimistic and eager to continue the trial and understand the full potential of OCU400.” Ocugen will continue to monitor long-term safety and efficacy data from the treated patients and provide additional updates. A webcast and conference call will take place today at 8:30 a.m. ET: Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers Conference ID: 7803227 Webcast: Available on the events section of the Ocugen investorsite AboutModifierGeneTherapy Modifier gene therapy is designed to fulfill unmet medical needs related to retinal diseases, including IRDs, such as RP, LCA, and Stargardt disease, as well as dry AMD. Our modifier gene therapy platform is based on the use of Nuclear hormone receptors (NHRs), master gene regulators, which have the potential to restore homeostasis — the basic biological processes in the retina. Unlike single-gene replacement therapies, which only target one genetic mutation, we believe that our modifier gene therapy platform, through its use of NHRs, represents a novel approach that has the potential to address multiple retinal diseases caused by mutations in multiple genes with one product, and to address complex diseases that are potentially caused by imbalances in multiple gene networks. Currently Ocugen has three modifier gene therapy programs OCU400 (RP, LCA), OCU410 (dry AMD), OCU410ST (Stargardt disease). AboutOCU400 OCU400 is the Company’s gene-agnostic modifier gene therapy product based on NHR gene, NR2E3. NR2E3 regulates diverse physiological functions within the retina—such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks. Through its drive functionality, OCU400 resets altered/affected cellular gene-networks and establishes homeostasis—a state of balance, which has the potential to improve retinal health and function in patients with inherited retinal diseases. AboutOcugen,Inc. Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patients’ lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at and follow us on Twitter and LinkedIn. CautionaryNoteonForward-LookingStatements Thispressreleasecontainsforward-lookingstatementswithinthemeaningofThePrivateSecuritiesLitigationReformActof1995,whicharesubject torisksanduncertainties,including,butnotlimitedto,statementsregardingqualitative assessments of available data, potential benefits, expectations for ongoing clinical trial results, and anticipated timing of clinical trial updates and regulatory interactions.Wemay,insomecases,usetermssuchas“predicts,”“believes,”“potential,”“proposed,”“continue,”“estimates,”“anticipates,”“expects,” “plans,”“intends,”“may,”“could,”“might,”“will,”“should,”orotherwordsthatconveyuncertaintyoffutureeventsoroutcomestoidentifytheseforward- lookingstatements.Suchstatementsaresubjecttonumerousimportantfactors,risks,anduncertaintiesthatmaycauseactualeventsorresultsto differmateriallyfromourcurrentexpectations,including,butnotlimitedto, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in the Phase 1/2 clinical trial or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities.TheseandotherrisksanduncertaintiesaremorefullydescribedinourperiodicfilingswiththeSecuritiesand ExchangeCommission(SEC),includingtheriskfactorsdescribedinthesectionentitled“RiskFactors”inthequarterlyandannualreportsthatwe theSEC.Anyforward-lookingstatementsthatwemakeinthispressreleasespeakonlyasofthedateofthispressrelease.Exceptasrequiredby law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release. Contact: Tiffany Hamilton Head of Communications Tiffany.Hamilton@ocugen.com

Phalguni Deswal @Phalguni_GD Ocugen announced that the manufacturing facility for its regenerative cell therapy for cartilage, NeoCart, is expected for completion by the end of 2023. Image Credit: Sergey Nivens / Shutterstock. Phase I/II trials for Ocugen ’s gene therapies, OCU410 and OCU410ST, are planned for initiation by the end of 2023 in geographic atrophy and Stargardt disease. The plans follow the US Food and Drug Administration’s (FDA) approval of the Investigational New Drug (IND) application for the two gene therapies. Recommended Reports Reports LOA and PTSR Model - Gene Therapy To Target Bcma and Cd19 in Waldenstrom Macroglobulinemia (Lymph... GlobalData Reports LOA and PTSR Model - Gene Therapy to Target BCMA and CS1 for Relapsed and Refractory Multiple Mye... GlobalData View all Companies Intelligence Ocugen Inc View all Ocugen is also planning for a Phase III trial in retinitis pigmentosa for OCU400 in Q4 2023/Q1 2024. The Phase III trial initiation is contingent on the FDA’s approval of trial plans and Phase I/II data. OCU410 is an adeno-associated virus containing a gene that encodes human retinoic acid receptor (RAR)-related orphan receptor alpha (RORα). It is planned to be investigated in geographic atrophy, which is the chronic degeneration of the macula during the late stages of age-related macular degeneration. OCU410ST was granted the orphan drug designation by the FDA for the treatment of Stargardt disease. Also known as ABCA4 retinopathy, the illness is a rare genetic disorder caused by pathogenic variations in the ABCA4 gene leading to fatty build-up on the macula and loss of vision. OCU400 is an adeno-associated virus serotype 5 capsid containing the gene for human nuclear hormone receptor NR2E3. It is being developed in partnership with China-based CanSinoBIO. It is currently being evaluated in an open-label Phase II trial (NCT05203939). An open-label Phase I trial (NCT05802329) for OCU200 in patients with diabetic macular oedema was initiated in Q2 2023. One of the cohorts in the trial will evaluate the combination therapy of OCU200 and Novartis ’ Lucentis (ranibizumab). Ocugen announced that the manufacturing facility for its regenerative cell therapy for cartilage, NeoCart, is expected for completion by the end of 2023, with a Phase III trial for the therapy planned for initiation in H2 2024. The US-based company is looking for government funding for its inhaled mucosal vaccine platform, with the IND application for the treatment expected to be filled in 2024. As part of their second quarter financials, Ocugen reported cash equivalents of $70.6m on 30 June 2023. The company also reported a loss of $0.10 per common share in Q2 2023. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. By Cytiva Thematic By downloading this case study, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy